2019 Future of Spinal Muscular Atrophy (SMA) R&D Pipeline Drugs and Companies- Analysis of Pipeline Compounds, Phases, Mechanism of Action, Clinical Trials and Developments
The global demand for Spinal Muscular Atrophy (SMA) treatment options is emerging rapidly driven by demand in major emerging and developed markets. An increasing number of companies are working on Spinal Muscular Atrophy (SMA) pipeline. Companies quickly adapting to the new technologies both independently and through collaborations and advancing their pipeline candidates to advanced phases will evolve as top performers. However, challenges of completing clinical trials on schedules and limited financial support are holding some of the Spinal Muscular Atrophy (SMA) pipeline companies from advancing their products into Phase 3 or Phase 4.
Spinal Muscular Atrophy (SMA) Report Description-
The 2019 pipeline study on Spinal Muscular Atrophy (SMA) pipeline is a comprehensive research on the drug candidates across different phases. This research study evaluates each of the pipeline products in terms of their current status, regulatory progress and expected phase completion date. Focusing on the factors driving and challenging pipeline drug development, this strategic analysis review presents detailed analysis on all active Spinal Muscular Atrophy (SMA) pipeline compounds.
The Spinal Muscular Atrophy (SMA) pipeline guide presents information on all active drugs currently being developed for Spinal Muscular Atrophy (SMA). The research work provides details of active therapeutic candidates in different phases including discovery, research, pre-clinical, phase I, Phase II, Phase III and pre-registration phases.
Details of current status, R&D progress and latest developments for every Spinal Muscular Atrophy (SMA) pipeline candidate are analyzed.
Further, current phase of development, mechanism of drug action, special designations such as orphan drug status, fast track designation, grants awarded, etc are presented for all Spinal Muscular Atrophy (SMA) drug candidates.
Drug development companies, collaborators, originating companies, license providers and universities participating in the Spinal Muscular Atrophy (SMA) product pipeline are provided. Company overview, snapshot and developments are provided for all participating companies.
The report assists in identifying potential upcoming companies and drugs in Spinal Muscular Atrophy (SMA) pipeline market along with progress of each drug. It also enables users to formulate effective strategies to stay ahead of competition.
VPA Research has developed a robust methodology for assessing pharmaceutical drugs, market sizes and sound forecast tools. All our research reports are provided through intense and repetitive primary and secondary research methods. Further, these reports are validated with industry experts to ensure reliability in the current scenario. The report is presented in a user-friendly format and presents clear and actionable insights.
Scope of Spinal Muscular Atrophy (SMA) pipeline report includes-
Spinal Muscular Atrophy (SMA) Report Description-
The 2019 pipeline study on Spinal Muscular Atrophy (SMA) pipeline is a comprehensive research on the drug candidates across different phases. This research study evaluates each of the pipeline products in terms of their current status, regulatory progress and expected phase completion date. Focusing on the factors driving and challenging pipeline drug development, this strategic analysis review presents detailed analysis on all active Spinal Muscular Atrophy (SMA) pipeline compounds.
The Spinal Muscular Atrophy (SMA) pipeline guide presents information on all active drugs currently being developed for Spinal Muscular Atrophy (SMA). The research work provides details of active therapeutic candidates in different phases including discovery, research, pre-clinical, phase I, Phase II, Phase III and pre-registration phases.
Details of current status, R&D progress and latest developments for every Spinal Muscular Atrophy (SMA) pipeline candidate are analyzed.
Further, current phase of development, mechanism of drug action, special designations such as orphan drug status, fast track designation, grants awarded, etc are presented for all Spinal Muscular Atrophy (SMA) drug candidates.
Drug development companies, collaborators, originating companies, license providers and universities participating in the Spinal Muscular Atrophy (SMA) product pipeline are provided. Company overview, snapshot and developments are provided for all participating companies.
The report assists in identifying potential upcoming companies and drugs in Spinal Muscular Atrophy (SMA) pipeline market along with progress of each drug. It also enables users to formulate effective strategies to stay ahead of competition.
VPA Research has developed a robust methodology for assessing pharmaceutical drugs, market sizes and sound forecast tools. All our research reports are provided through intense and repetitive primary and secondary research methods. Further, these reports are validated with industry experts to ensure reliability in the current scenario. The report is presented in a user-friendly format and presents clear and actionable insights.
Scope of Spinal Muscular Atrophy (SMA) pipeline report includes-
- An overview of Spinal Muscular Atrophy (SMA) disease including symptoms, causes, diagnosis and available treatment options is provided.
- Snapshot of pipeline detailing number of active compounds, mergers and acquisitions, phase wise compounds, target areas, mechanism of action, trials etc
- Phase wise count of Spinal Muscular Atrophy (SMA) pipeline
- Company wise list of Spinal Muscular Atrophy (SMA) pipeline
- Mechanism of Action wise Spinal Muscular Atrophy (SMA) pipeline
- For each pipeline candidate, the following details are provided
- Snapshot (Drug, Mechanism of Action, Phase, Co Developer, Originator, Synonym, Orphan Drug Status, New Molecular Entity, Area)
- Current status of development
- Drug overview
- Mechanism of Action
- Pre-clinical and Clinical Trials/Results
- Company Overview and Recent Developments
- The report is designed to help industry executives promote the success and continued growth of their organizations
- Get clear understanding of the entire Spinal Muscular Atrophy (SMA) pipeline, with details on active projects
- Stay ahead of the competition through comprehensive knowledge of Spinal Muscular Atrophy (SMA) pipeline progress
- Get in detail information of each product with updated information on each project along with key milestones
- Gain clear insights into companies participating in Spinal Muscular Atrophy (SMA) pipeline along with progress of each of the company’s products and information on associations/collaborations/licensing data
- Get trial information for each pipeline product under development
- Understand the pipeline structure in terms of mechanism of Action, phase and company
1. TABLE OF CONTENTS
1.1 List of Tables
1.2 List of Figures
2. GLOBAL SPINAL MUSCULAR ATROPHY (SMA) PIPELINE OVERVIEW
2.1 Key Findings, 2019
2.2 Disease Overview
2.3 Research Methodology
3. EXECUTIVE SUMMARY
3.1 Spinal Muscular Atrophy (SMA) Drugs under active development, H1- 2019
3.2 Pipeline Drugs in Early Stage of Development
3.2.1 Pre-clinical
3.2.2 Discovery
3.2.3 Phase
3.2.4Phase
3.3 Pipeline Drugs in Advanced Stage of Development
3.3.1 Phase
3.3.2 Pre-registration
3.4 Companies involved in Spinal Muscular Atrophy (SMA) pipeline, H1- 2019
3.5 Mechanism of Action wise Spinal Muscular Atrophy (SMA) Pipeline Candidates
4 ANDROSCIENCE CORP SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
4.1 AndroScience Corp Business Profile
4.2 AndroScience Corp Spinal Muscular Atrophy (SMA) Drug Details
4.3 Drug Snapshot
4.3.1 Originator
4.3.2 Collaborator/Co-Developer
4.3.3 Route of Administration
4.3.4 Orphan Drug/Fast Track/Special Designation
4.3.5 Geography
4.3.6 Type of Molecular Entity
4.3.7 Current Status
4.4 Drug Overview
4.5 Drug Mechanism of Action
4.6 Clinical/Pre-clinical Trial Details
4.7 Latest Drug Developments
5 ARMGO PHARMA INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
5.1 Armgo Pharma Inc Business Profile
5.2 Armgo Pharma Inc Spinal Muscular Atrophy (SMA) Drug Details
5.3 Drug Snapshot
5.3.1 Originator
5.3.2 Collaborator/Co-Developer
5.3.3 Route of Administration
5.3.4 Orphan Drug/Fast Track/Special Designation
5.3.5 Geography
5.3.6 Type of Molecular Entity
5.3.7 Current Status
5.4 Drug Overview
5.5 Drug Mechanism of Action
5.6 Clinical/Pre-clinical Trial Details
5.7 Latest Drug Developments
6 AURIMMED PHARMA INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
6.1 AurimMed Pharma Inc Business Profile
6.2 AurimMed Pharma Inc Spinal Muscular Atrophy (SMA) Drug Details
6.3 Drug Snapshot
6.3.1 Originator
6.3.2 Collaborator/Co-Developer
6.3.3 Route of Administration
6.3.4 Orphan Drug/Fast Track/Special Designation
6.3.5 Geography
6.3.6 Type of Molecular Entity
6.3.7 Current Status
6.4 Drug Overview
6.5 Drug Mechanism of Action
6.6 Clinical/Pre-clinical Trial Details
6.7 Latest Drug Developments
7 AVEXIS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
7.1 AveXis Inc Business Profile
7.2 AveXis Inc Spinal Muscular Atrophy (SMA) Drug Details
7.3 Drug Snapshot
7.3.1 Originator
7.3.2 Collaborator/Co-Developer
7.3.3 Route of Administration
7.3.4 Orphan Drug/Fast Track/Special Designation
7.3.5 Geography
7.3.6 Type of Molecular Entity
7.3.7 Current Status
7.4 Drug Overview
7.5 Drug Mechanism of Action
7.6 Clinical/Pre-clinical Trial Details
7.7 Latest Drug Developments
8 BIOGEN INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
8.1 Biogen Inc Business Profile
8.2 Biogen Inc Spinal Muscular Atrophy (SMA) Drug Details
8.3 Drug Snapshot
8.3.1 Originator
8.3.2 Collaborator/Co-Developer
8.3.3 Route of Administration
8.3.4 Orphan Drug/Fast Track/Special Designation
8.3.5 Geography
8.3.6 Type of Molecular Entity
8.3.7 Current Status
8.4 Drug Overview
8.5 Drug Mechanism of Action
8.6 Clinical/Pre-clinical Trial Details
8.7 Latest Drug Developments
9 BIOMARIN PHARMACEUTICAL INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
9.1 BioMarin Pharmaceutical Inc Business Profile
9.2 BioMarin Pharmaceutical Inc Spinal Muscular Atrophy (SMA) Drug Details
9.3 Drug Snapshot
9.3.1 Originator
9.3.2 Collaborator/Co-Developer
9.3.3 Route of Administration
9.3.4 Orphan Drug/Fast Track/Special Designation
9.3.5 Geography
9.3.6 Type of Molecular Entity
9.3.7 Current Status
9.4 Drug Overview
9.5 Drug Mechanism of Action
9.6 Clinical/Pre-clinical Trial Details
9.7 Latest Drug Developments
10 CYTOKINETICS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
10.1 Cytokinetics Inc Business Profile
10.2 Cytokinetics Inc Spinal Muscular Atrophy (SMA) Drug Details
10.3 Drug Snapshot
10.3.1 Originator
10.3.2 Collaborator/Co-Developer
10.3.3 Route of Administration
10.3.4 Orphan Drug/Fast Track/Special Designation
10.3.5 Geography
10.3.6 Type of Molecular Entity
10.3.7 Current Status
10.4 Drug Overview
10.5 Drug Mechanism of Action
10.6 Clinical/Pre-clinical Trial Details
10.7 Latest Drug Developments
11 EXICURE INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
11.1 Exicure Inc Business Profile
11.2 Exicure Inc Spinal Muscular Atrophy (SMA) Drug Details
11.3 Drug Snapshot
11.3.1 Originator
11.3.2 Collaborator/Co-Developer
11.3.3 Route of Administration
11.3.4 Orphan Drug/Fast Track/Special Designation
11.3.5 Geography
11.3.6 Type of Molecular Entity
11.3.7 Current Status
11.4 Drug Overview
11.5 Drug Mechanism of Action
11.6 Clinical/Pre-clinical Trial Details
11.7 Latest Drug Developments
12 F. HOFFMANN-LA ROCHE LTD SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
12.1 F. Hoffmann-La Roche Ltd Business Profile
12.2 F. Hoffmann-La Roche Ltd Spinal Muscular Atrophy (SMA) Drug Details
12.3 Drug Snapshot
12.3.1 Originator
12.3.2 Collaborator/Co-Developer
12.3.3 Route of Administration
12.3.4 Orphan Drug/Fast Track/Special Designation
12.3.5 Geography
12.3.6 Type of Molecular Entity
12.3.7 Current Status
12.4 Drug Overview
12.5 Drug Mechanism of Action
12.6 Clinical/Pre-clinical Trial Details
12.7 Latest Drug Developments
13 GENEA BIOCELLS SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
13.1 Genea Biocells Business Profile
13.2 Genea Biocells Spinal Muscular Atrophy (SMA) Drug Details
13.3 Drug Snapshot
13.3.1 Originator
13.3.2 Collaborator/Co-Developer
13.3.3 Route of Administration
13.3.4 Orphan Drug/Fast Track/Special Designation
13.3.5 Geography
13.3.6 Type of Molecular Entity
13.3.7 Current Status
13.4 Drug Overview
13.5 Drug Mechanism of Action
13.6 Clinical/Pre-clinical Trial Details
13.7 Latest Drug Developments
14 GENETHON SA SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
14.1 Genethon SA Business Profile
14.2 Genethon SA Spinal Muscular Atrophy (SMA) Drug Details
14.3 Drug Snapshot
14.3.1 Originator
14.3.2 Collaborator/Co-Developer
14.3.3 Route of Administration
14.3.4 Orphan Drug/Fast Track/Special Designation
14.3.5 Geography
14.3.6 Type of Molecular Entity
14.3.7 Current Status
14.4 Drug Overview
14.5 Drug Mechanism of Action
14.6 Clinical/Pre-clinical Trial Details
14.7 Latest Drug Developments
15 GNT PHARMA CO LTD SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
15.1 GNT Pharma Co Ltd Business Profile
15.2 GNT Pharma Co Ltd Spinal Muscular Atrophy (SMA) Drug Details
15.3 Drug Snapshot
15.3.1 Originator
15.3.2 Collaborator/Co-Developer
15.3.3 Route of Administration
15.3.4 Orphan Drug/Fast Track/Special Designation
15.3.5 Geography
15.3.6 Type of Molecular Entity
15.3.7 Current Status
15.4 Drug Overview
15.5 Drug Mechanism of Action
15.6 Clinical/Pre-clinical Trial Details
15.7 Latest Drug Developments
16 KOWA CO LTD SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
16.1 Kowa Co Ltd Business Profile
16.2 Kowa Co Ltd Spinal Muscular Atrophy (SMA) Drug Details
16.3 Drug Snapshot
16.3.1 Originator
16.3.2 Collaborator/Co-Developer
16.3.3 Route of Administration
16.3.4 Orphan Drug/Fast Track/Special Designation
16.3.5 Geography
16.3.6 Type of Molecular Entity
16.3.7 Current Status
16.4 Drug Overview
16.5 Drug Mechanism of Action
16.6 Clinical/Pre-clinical Trial Details
16.7 Latest Drug Developments
17 NEURODYN LIFE SCIENCES INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
17.1 Neurodyn Life Sciences Inc Business Profile
17.2 Neurodyn Life Sciences Inc Spinal Muscular Atrophy (SMA) Drug Details
17.3 Drug Snapshot
17.3.1 Originator
17.3.2 Collaborator/Co-Developer
17.3.3 Route of Administration
17.3.4 Orphan Drug/Fast Track/Special Designation
17.3.5 Geography
17.3.6 Type of Molecular Entity
17.3.7 Current Status
17.4 Drug Overview
17.5 Drug Mechanism of Action
17.6 Clinical/Pre-clinical Trial Details
17.7 Latest Drug Developments
18 NEUROTUNE AG SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
18.1 Neurotune AG Business Profile
18.2 Neurotune AG Spinal Muscular Atrophy (SMA) Drug Details
18.3 Drug Snapshot
18.3.1 Originator
18.3.2 Collaborator/Co-Developer
18.3.3 Route of Administration
18.3.4 Orphan Drug/Fast Track/Special Designation
18.3.5 Geography
18.3.6 Type of Molecular Entity
18.3.7 Current Status
18.4 Drug Overview
18.5 Drug Mechanism of Action
18.6 Clinical/Pre-clinical Trial Details
18.7 Latest Drug Developments
19 NOVARTIS AG SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
19.1 Novartis AG Business Profile
19.2 Novartis AG Spinal Muscular Atrophy (SMA) Drug Details
19.3 Drug Snapshot
19.3.1 Originator
19.3.2 Collaborator/Co-Developer
19.3.3 Route of Administration
19.3.4 Orphan Drug/Fast Track/Special Designation
19.3.5 Geography
19.3.6 Type of Molecular Entity
19.3.7 Current Status
19.4 Drug Overview
19.5 Drug Mechanism of Action
19.6 Clinical/Pre-clinical Trial Details
19.7 Latest Drug Developments
20 ONO PHARMACEUTICAL CO LTD SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
20.1 Ono Pharmaceutical Co Ltd Business Profile
20.2 Ono Pharmaceutical Co Ltd Spinal Muscular Atrophy (SMA) Drug Details
20.3 Drug Snapshot
20.3.1 Originator
20.3.2 Collaborator/Co-Developer
20.3.3 Route of Administration
20.3.4 Orphan Drug/Fast Track/Special Designation
20.3.5 Geography
20.3.6 Type of Molecular Entity
20.3.7 Current Status
20.4 Drug Overview
20.5 Drug Mechanism of Action
20.6 Clinical/Pre-clinical Trial Details
20.7 Latest Drug Developments
21 PARATEK PHARMACEUTICALS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
21.1 Paratek Pharmaceuticals Inc Business Profile
21.2 Paratek Pharmaceuticals Inc Spinal Muscular Atrophy (SMA) Drug Details
21.3 Drug Snapshot
21.3.1 Originator
21.3.2 Collaborator/Co-Developer
21.3.3 Route of Administration
21.3.4 Orphan Drug/Fast Track/Special Designation
21.3.5 Geography
21.3.6 Type of Molecular Entity
21.3.7 Current Status
21.4 Drug Overview
21.5 Drug Mechanism of Action
21.6 Clinical/Pre-clinical Trial Details
21.7 Latest Drug Developments
22 REBORNA BIOSCIENCES INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
22.1 Reborna Biosciences Inc Business Profile
22.2 Reborna Biosciences Inc Spinal Muscular Atrophy (SMA) Drug Details
22.3 Drug Snapshot
22.3.1 Originator
22.3.2 Collaborator/Co-Developer
22.3.3 Route of Administration
22.3.4 Orphan Drug/Fast Track/Special Designation
22.3.5 Geography
22.3.6 Type of Molecular Entity
22.3.7 Current Status
22.4 Drug Overview
22.5 Drug Mechanism of Action
22.6 Clinical/Pre-clinical Trial Details
22.7 Latest Drug Developments
23 RECURSION PHARMACEUTICALS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
23.1 Recursion Pharmaceuticals Inc Business Profile
23.2 Recursion Pharmaceuticals Inc Spinal Muscular Atrophy (SMA) Drug Details
23.3 Drug Snapshot
23.3.1 Originator
23.3.2 Collaborator/Co-Developer
23.3.3 Route of Administration
23.3.4 Orphan Drug/Fast Track/Special Designation
23.3.5 Geography
23.3.6 Type of Molecular Entity
23.3.7 Current Status
23.4 Drug Overview
23.5 Drug Mechanism of Action
23.6 Clinical/Pre-clinical Trial Details
23.7 Latest Drug Developments
24 SAREPTA THERAPEUTICS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
24.1 Sarepta Therapeutics Inc Business Profile
24.2 Sarepta Therapeutics Inc Spinal Muscular Atrophy (SMA) Drug Details
24.3 Drug Snapshot
24.3.1 Originator
24.3.2 Collaborator/Co-Developer
24.3.3 Route of Administration
24.3.4 Orphan Drug/Fast Track/Special Designation
24.3.5 Geography
24.3.6 Type of Molecular Entity
24.3.7 Current Status
24.4 Drug Overview
24.5 Drug Mechanism of Action
24.6 Clinical/Pre-clinical Trial Details
24.7 Latest Drug Developments
25 SCHOLAR ROCK INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
25.1 Scholar Rock Inc Business Profile
25.2 Scholar Rock Inc Spinal Muscular Atrophy (SMA) Drug Details
25.3 Drug Snapshot
25.3.1 Originator
25.3.2 Collaborator/Co-Developer
25.3.3 Route of Administration
25.3.4 Orphan Drug/Fast Track/Special Designation
25.3.5 Geography
25.3.6 Type of Molecular Entity
25.3.7 Current Status
25.4 Drug Overview
25.5 Drug Mechanism of Action
25.6 Clinical/Pre-clinical Trial Details
25.7 Latest Drug Developments
26 SHIFT PHARMACEUTICALS SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
26.1 Shift Pharmaceuticals Business Profile
26.2 Shift Pharmaceuticals Spinal Muscular Atrophy (SMA) Drug Details
26.3 Drug Snapshot
26.3.1 Originator
26.3.2 Collaborator/Co-Developer
26.3.3 Route of Administration
26.3.4 Orphan Drug/Fast Track/Special Designation
26.3.5 Geography
26.3.6 Type of Molecular Entity
26.3.7 Current Status
26.4 Drug Overview
26.5 Drug Mechanism of Action
26.6 Clinical/Pre-clinical Trial Details
26.7 Latest Drug Developments
27 SPOTLIGHT INNOVATION INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
27.1 Spotlight Innovation Inc Business Profile
27.2 Spotlight Innovation Inc Spinal Muscular Atrophy (SMA) Drug Details
27.3 Drug Snapshot
27.3.1 Originator
27.3.2 Collaborator/Co-Developer
27.3.3 Route of Administration
27.3.4 Orphan Drug/Fast Track/Special Designation
27.3.5 Geography
27.3.6 Type of Molecular Entity
27.3.7 Current Status
27.4 Drug Overview
27.5 Drug Mechanism of Action
27.6 Clinical/Pre-clinical Trial Details
27.7 Latest Drug Developments
28 VYBION INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
28.1 Vybion Inc Business Profile
28.2 Vybion Inc Spinal Muscular Atrophy (SMA) Drug Details
28.3 Drug Snapshot
28.3.1 Originator
28.3.2 Collaborator/Co-Developer
28.3.3 Route of Administration
28.3.4 Orphan Drug/Fast Track/Special Designation
28.3.5 Geography
28.3.6 Type of Molecular Entity
28.3.7 Current Status
28.4 Drug Overview
28.5 Drug Mechanism of Action
28.6 Clinical/Pre-clinical Trial Details
28.7 Latest Drug Developments
29. LATEST SPINAL MUSCULAR ATROPHY (SMA) DRUG PIPELINE DEVELOPMENTS, 2019
30. APPENDIX
30.1 About Us
30.2 Sources and Methodology
30.3 Contact Information
1.1 List of Tables
1.2 List of Figures
2. GLOBAL SPINAL MUSCULAR ATROPHY (SMA) PIPELINE OVERVIEW
2.1 Key Findings, 2019
2.2 Disease Overview
2.3 Research Methodology
3. EXECUTIVE SUMMARY
3.1 Spinal Muscular Atrophy (SMA) Drugs under active development, H1- 2019
3.2 Pipeline Drugs in Early Stage of Development
3.2.1 Pre-clinical
3.2.2 Discovery
3.2.3 Phase
3.2.4Phase
3.3 Pipeline Drugs in Advanced Stage of Development
3.3.1 Phase
3.3.2 Pre-registration
3.4 Companies involved in Spinal Muscular Atrophy (SMA) pipeline, H1- 2019
3.5 Mechanism of Action wise Spinal Muscular Atrophy (SMA) Pipeline Candidates
4 ANDROSCIENCE CORP SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
4.1 AndroScience Corp Business Profile
4.2 AndroScience Corp Spinal Muscular Atrophy (SMA) Drug Details
4.3 Drug Snapshot
4.3.1 Originator
4.3.2 Collaborator/Co-Developer
4.3.3 Route of Administration
4.3.4 Orphan Drug/Fast Track/Special Designation
4.3.5 Geography
4.3.6 Type of Molecular Entity
4.3.7 Current Status
4.4 Drug Overview
4.5 Drug Mechanism of Action
4.6 Clinical/Pre-clinical Trial Details
4.7 Latest Drug Developments
5 ARMGO PHARMA INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
5.1 Armgo Pharma Inc Business Profile
5.2 Armgo Pharma Inc Spinal Muscular Atrophy (SMA) Drug Details
5.3 Drug Snapshot
5.3.1 Originator
5.3.2 Collaborator/Co-Developer
5.3.3 Route of Administration
5.3.4 Orphan Drug/Fast Track/Special Designation
5.3.5 Geography
5.3.6 Type of Molecular Entity
5.3.7 Current Status
5.4 Drug Overview
5.5 Drug Mechanism of Action
5.6 Clinical/Pre-clinical Trial Details
5.7 Latest Drug Developments
6 AURIMMED PHARMA INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
6.1 AurimMed Pharma Inc Business Profile
6.2 AurimMed Pharma Inc Spinal Muscular Atrophy (SMA) Drug Details
6.3 Drug Snapshot
6.3.1 Originator
6.3.2 Collaborator/Co-Developer
6.3.3 Route of Administration
6.3.4 Orphan Drug/Fast Track/Special Designation
6.3.5 Geography
6.3.6 Type of Molecular Entity
6.3.7 Current Status
6.4 Drug Overview
6.5 Drug Mechanism of Action
6.6 Clinical/Pre-clinical Trial Details
6.7 Latest Drug Developments
7 AVEXIS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
7.1 AveXis Inc Business Profile
7.2 AveXis Inc Spinal Muscular Atrophy (SMA) Drug Details
7.3 Drug Snapshot
7.3.1 Originator
7.3.2 Collaborator/Co-Developer
7.3.3 Route of Administration
7.3.4 Orphan Drug/Fast Track/Special Designation
7.3.5 Geography
7.3.6 Type of Molecular Entity
7.3.7 Current Status
7.4 Drug Overview
7.5 Drug Mechanism of Action
7.6 Clinical/Pre-clinical Trial Details
7.7 Latest Drug Developments
8 BIOGEN INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
8.1 Biogen Inc Business Profile
8.2 Biogen Inc Spinal Muscular Atrophy (SMA) Drug Details
8.3 Drug Snapshot
8.3.1 Originator
8.3.2 Collaborator/Co-Developer
8.3.3 Route of Administration
8.3.4 Orphan Drug/Fast Track/Special Designation
8.3.5 Geography
8.3.6 Type of Molecular Entity
8.3.7 Current Status
8.4 Drug Overview
8.5 Drug Mechanism of Action
8.6 Clinical/Pre-clinical Trial Details
8.7 Latest Drug Developments
9 BIOMARIN PHARMACEUTICAL INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
9.1 BioMarin Pharmaceutical Inc Business Profile
9.2 BioMarin Pharmaceutical Inc Spinal Muscular Atrophy (SMA) Drug Details
9.3 Drug Snapshot
9.3.1 Originator
9.3.2 Collaborator/Co-Developer
9.3.3 Route of Administration
9.3.4 Orphan Drug/Fast Track/Special Designation
9.3.5 Geography
9.3.6 Type of Molecular Entity
9.3.7 Current Status
9.4 Drug Overview
9.5 Drug Mechanism of Action
9.6 Clinical/Pre-clinical Trial Details
9.7 Latest Drug Developments
10 CYTOKINETICS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
10.1 Cytokinetics Inc Business Profile
10.2 Cytokinetics Inc Spinal Muscular Atrophy (SMA) Drug Details
10.3 Drug Snapshot
10.3.1 Originator
10.3.2 Collaborator/Co-Developer
10.3.3 Route of Administration
10.3.4 Orphan Drug/Fast Track/Special Designation
10.3.5 Geography
10.3.6 Type of Molecular Entity
10.3.7 Current Status
10.4 Drug Overview
10.5 Drug Mechanism of Action
10.6 Clinical/Pre-clinical Trial Details
10.7 Latest Drug Developments
11 EXICURE INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
11.1 Exicure Inc Business Profile
11.2 Exicure Inc Spinal Muscular Atrophy (SMA) Drug Details
11.3 Drug Snapshot
11.3.1 Originator
11.3.2 Collaborator/Co-Developer
11.3.3 Route of Administration
11.3.4 Orphan Drug/Fast Track/Special Designation
11.3.5 Geography
11.3.6 Type of Molecular Entity
11.3.7 Current Status
11.4 Drug Overview
11.5 Drug Mechanism of Action
11.6 Clinical/Pre-clinical Trial Details
11.7 Latest Drug Developments
12 F. HOFFMANN-LA ROCHE LTD SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
12.1 F. Hoffmann-La Roche Ltd Business Profile
12.2 F. Hoffmann-La Roche Ltd Spinal Muscular Atrophy (SMA) Drug Details
12.3 Drug Snapshot
12.3.1 Originator
12.3.2 Collaborator/Co-Developer
12.3.3 Route of Administration
12.3.4 Orphan Drug/Fast Track/Special Designation
12.3.5 Geography
12.3.6 Type of Molecular Entity
12.3.7 Current Status
12.4 Drug Overview
12.5 Drug Mechanism of Action
12.6 Clinical/Pre-clinical Trial Details
12.7 Latest Drug Developments
13 GENEA BIOCELLS SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
13.1 Genea Biocells Business Profile
13.2 Genea Biocells Spinal Muscular Atrophy (SMA) Drug Details
13.3 Drug Snapshot
13.3.1 Originator
13.3.2 Collaborator/Co-Developer
13.3.3 Route of Administration
13.3.4 Orphan Drug/Fast Track/Special Designation
13.3.5 Geography
13.3.6 Type of Molecular Entity
13.3.7 Current Status
13.4 Drug Overview
13.5 Drug Mechanism of Action
13.6 Clinical/Pre-clinical Trial Details
13.7 Latest Drug Developments
14 GENETHON SA SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
14.1 Genethon SA Business Profile
14.2 Genethon SA Spinal Muscular Atrophy (SMA) Drug Details
14.3 Drug Snapshot
14.3.1 Originator
14.3.2 Collaborator/Co-Developer
14.3.3 Route of Administration
14.3.4 Orphan Drug/Fast Track/Special Designation
14.3.5 Geography
14.3.6 Type of Molecular Entity
14.3.7 Current Status
14.4 Drug Overview
14.5 Drug Mechanism of Action
14.6 Clinical/Pre-clinical Trial Details
14.7 Latest Drug Developments
15 GNT PHARMA CO LTD SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
15.1 GNT Pharma Co Ltd Business Profile
15.2 GNT Pharma Co Ltd Spinal Muscular Atrophy (SMA) Drug Details
15.3 Drug Snapshot
15.3.1 Originator
15.3.2 Collaborator/Co-Developer
15.3.3 Route of Administration
15.3.4 Orphan Drug/Fast Track/Special Designation
15.3.5 Geography
15.3.6 Type of Molecular Entity
15.3.7 Current Status
15.4 Drug Overview
15.5 Drug Mechanism of Action
15.6 Clinical/Pre-clinical Trial Details
15.7 Latest Drug Developments
16 KOWA CO LTD SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
16.1 Kowa Co Ltd Business Profile
16.2 Kowa Co Ltd Spinal Muscular Atrophy (SMA) Drug Details
16.3 Drug Snapshot
16.3.1 Originator
16.3.2 Collaborator/Co-Developer
16.3.3 Route of Administration
16.3.4 Orphan Drug/Fast Track/Special Designation
16.3.5 Geography
16.3.6 Type of Molecular Entity
16.3.7 Current Status
16.4 Drug Overview
16.5 Drug Mechanism of Action
16.6 Clinical/Pre-clinical Trial Details
16.7 Latest Drug Developments
17 NEURODYN LIFE SCIENCES INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
17.1 Neurodyn Life Sciences Inc Business Profile
17.2 Neurodyn Life Sciences Inc Spinal Muscular Atrophy (SMA) Drug Details
17.3 Drug Snapshot
17.3.1 Originator
17.3.2 Collaborator/Co-Developer
17.3.3 Route of Administration
17.3.4 Orphan Drug/Fast Track/Special Designation
17.3.5 Geography
17.3.6 Type of Molecular Entity
17.3.7 Current Status
17.4 Drug Overview
17.5 Drug Mechanism of Action
17.6 Clinical/Pre-clinical Trial Details
17.7 Latest Drug Developments
18 NEUROTUNE AG SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
18.1 Neurotune AG Business Profile
18.2 Neurotune AG Spinal Muscular Atrophy (SMA) Drug Details
18.3 Drug Snapshot
18.3.1 Originator
18.3.2 Collaborator/Co-Developer
18.3.3 Route of Administration
18.3.4 Orphan Drug/Fast Track/Special Designation
18.3.5 Geography
18.3.6 Type of Molecular Entity
18.3.7 Current Status
18.4 Drug Overview
18.5 Drug Mechanism of Action
18.6 Clinical/Pre-clinical Trial Details
18.7 Latest Drug Developments
19 NOVARTIS AG SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
19.1 Novartis AG Business Profile
19.2 Novartis AG Spinal Muscular Atrophy (SMA) Drug Details
19.3 Drug Snapshot
19.3.1 Originator
19.3.2 Collaborator/Co-Developer
19.3.3 Route of Administration
19.3.4 Orphan Drug/Fast Track/Special Designation
19.3.5 Geography
19.3.6 Type of Molecular Entity
19.3.7 Current Status
19.4 Drug Overview
19.5 Drug Mechanism of Action
19.6 Clinical/Pre-clinical Trial Details
19.7 Latest Drug Developments
20 ONO PHARMACEUTICAL CO LTD SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
20.1 Ono Pharmaceutical Co Ltd Business Profile
20.2 Ono Pharmaceutical Co Ltd Spinal Muscular Atrophy (SMA) Drug Details
20.3 Drug Snapshot
20.3.1 Originator
20.3.2 Collaborator/Co-Developer
20.3.3 Route of Administration
20.3.4 Orphan Drug/Fast Track/Special Designation
20.3.5 Geography
20.3.6 Type of Molecular Entity
20.3.7 Current Status
20.4 Drug Overview
20.5 Drug Mechanism of Action
20.6 Clinical/Pre-clinical Trial Details
20.7 Latest Drug Developments
21 PARATEK PHARMACEUTICALS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
21.1 Paratek Pharmaceuticals Inc Business Profile
21.2 Paratek Pharmaceuticals Inc Spinal Muscular Atrophy (SMA) Drug Details
21.3 Drug Snapshot
21.3.1 Originator
21.3.2 Collaborator/Co-Developer
21.3.3 Route of Administration
21.3.4 Orphan Drug/Fast Track/Special Designation
21.3.5 Geography
21.3.6 Type of Molecular Entity
21.3.7 Current Status
21.4 Drug Overview
21.5 Drug Mechanism of Action
21.6 Clinical/Pre-clinical Trial Details
21.7 Latest Drug Developments
22 REBORNA BIOSCIENCES INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
22.1 Reborna Biosciences Inc Business Profile
22.2 Reborna Biosciences Inc Spinal Muscular Atrophy (SMA) Drug Details
22.3 Drug Snapshot
22.3.1 Originator
22.3.2 Collaborator/Co-Developer
22.3.3 Route of Administration
22.3.4 Orphan Drug/Fast Track/Special Designation
22.3.5 Geography
22.3.6 Type of Molecular Entity
22.3.7 Current Status
22.4 Drug Overview
22.5 Drug Mechanism of Action
22.6 Clinical/Pre-clinical Trial Details
22.7 Latest Drug Developments
23 RECURSION PHARMACEUTICALS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
23.1 Recursion Pharmaceuticals Inc Business Profile
23.2 Recursion Pharmaceuticals Inc Spinal Muscular Atrophy (SMA) Drug Details
23.3 Drug Snapshot
23.3.1 Originator
23.3.2 Collaborator/Co-Developer
23.3.3 Route of Administration
23.3.4 Orphan Drug/Fast Track/Special Designation
23.3.5 Geography
23.3.6 Type of Molecular Entity
23.3.7 Current Status
23.4 Drug Overview
23.5 Drug Mechanism of Action
23.6 Clinical/Pre-clinical Trial Details
23.7 Latest Drug Developments
24 SAREPTA THERAPEUTICS INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
24.1 Sarepta Therapeutics Inc Business Profile
24.2 Sarepta Therapeutics Inc Spinal Muscular Atrophy (SMA) Drug Details
24.3 Drug Snapshot
24.3.1 Originator
24.3.2 Collaborator/Co-Developer
24.3.3 Route of Administration
24.3.4 Orphan Drug/Fast Track/Special Designation
24.3.5 Geography
24.3.6 Type of Molecular Entity
24.3.7 Current Status
24.4 Drug Overview
24.5 Drug Mechanism of Action
24.6 Clinical/Pre-clinical Trial Details
24.7 Latest Drug Developments
25 SCHOLAR ROCK INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
25.1 Scholar Rock Inc Business Profile
25.2 Scholar Rock Inc Spinal Muscular Atrophy (SMA) Drug Details
25.3 Drug Snapshot
25.3.1 Originator
25.3.2 Collaborator/Co-Developer
25.3.3 Route of Administration
25.3.4 Orphan Drug/Fast Track/Special Designation
25.3.5 Geography
25.3.6 Type of Molecular Entity
25.3.7 Current Status
25.4 Drug Overview
25.5 Drug Mechanism of Action
25.6 Clinical/Pre-clinical Trial Details
25.7 Latest Drug Developments
26 SHIFT PHARMACEUTICALS SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
26.1 Shift Pharmaceuticals Business Profile
26.2 Shift Pharmaceuticals Spinal Muscular Atrophy (SMA) Drug Details
26.3 Drug Snapshot
26.3.1 Originator
26.3.2 Collaborator/Co-Developer
26.3.3 Route of Administration
26.3.4 Orphan Drug/Fast Track/Special Designation
26.3.5 Geography
26.3.6 Type of Molecular Entity
26.3.7 Current Status
26.4 Drug Overview
26.5 Drug Mechanism of Action
26.6 Clinical/Pre-clinical Trial Details
26.7 Latest Drug Developments
27 SPOTLIGHT INNOVATION INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
27.1 Spotlight Innovation Inc Business Profile
27.2 Spotlight Innovation Inc Spinal Muscular Atrophy (SMA) Drug Details
27.3 Drug Snapshot
27.3.1 Originator
27.3.2 Collaborator/Co-Developer
27.3.3 Route of Administration
27.3.4 Orphan Drug/Fast Track/Special Designation
27.3.5 Geography
27.3.6 Type of Molecular Entity
27.3.7 Current Status
27.4 Drug Overview
27.5 Drug Mechanism of Action
27.6 Clinical/Pre-clinical Trial Details
27.7 Latest Drug Developments
28 VYBION INC SPINAL MUSCULAR ATROPHY (SMA) PIPELINE DETAILS
28.1 Vybion Inc Business Profile
28.2 Vybion Inc Spinal Muscular Atrophy (SMA) Drug Details
28.3 Drug Snapshot
28.3.1 Originator
28.3.2 Collaborator/Co-Developer
28.3.3 Route of Administration
28.3.4 Orphan Drug/Fast Track/Special Designation
28.3.5 Geography
28.3.6 Type of Molecular Entity
28.3.7 Current Status
28.4 Drug Overview
28.5 Drug Mechanism of Action
28.6 Clinical/Pre-clinical Trial Details
28.7 Latest Drug Developments
29. LATEST SPINAL MUSCULAR ATROPHY (SMA) DRUG PIPELINE DEVELOPMENTS, 2019
30. APPENDIX
30.1 About Us
30.2 Sources and Methodology
30.3 Contact Information
